By Robyn Marshall
A wide-ranging patent granted in the US at the end of March to the National Institutes of Health, a major government-funded research body, will cripple the development of life-saving gene therapies.
NIH has given the patent to a private company, GTI, of Gaithersburg, Maryland. Scientists were incredulous at the extent of the patent, which was applied for in the late 1980s.
The patent covers the broad principle of removing cells from a patient with a genetically caused disease, altering the gene responsible and returning the cells to the body. Almost every gene therapy technique that has been approved has used this technique. The patent gives GTI the exclusive right to develop the technique commercially.
In the last few years, biochemical research, and in particular molecular genetics, has mushroomed. Virtually every disease that is caused by a genetic error has been pinpointed and identified exactly. Sometimes it is only one base change in the DNA that results in a different amino acid being incorporated in the protein chain of maybe 500 amino acids.
Scientists are able to make millions of copies of the correct gene. It is also possible to cut out the correct gene and insert it into a piece of DNA. The central problem now is how to insert the correct gene into the affected chromosome so that the body's own cell nuclei in the appropriate organ make the correct protein.
In 1990, Anderson and Rosenberg carried out the first test of this type of gene therapy on a girl suffering from adenosine deaminase deficiency, in which the defective gene makes an inefficient enzyme and leaves the sufferer unable to fight infection. Anderson and Rosenberg inserted the correct version of the gene into the girl's white blood cells so that she could produce the enzyme herself.
Rival companies that develop therapies for other diseases based on altering cellular DNA in the laboratory before returning the cells to the body will now have to pay a licence fee to GTI or ditch the work altogether. It means there will be less funds available from private companies to fund this type of research
The patent does not apply to academic research, but to the application of the research. The continuation of research that is more advanced could depend on how much GTI charges in licence fees.
Many researchers say that the research wasn't a new idea. The idea of removing cells, modifying the DNA and returning them to the body was around for ages, and many experiments had been done on animals. It was the obvious step to carry out the experiment in humans. It was just that Anderson and Rosenberg were the first to do it.
Patients with any one of the genetic diseases will pay the price of the patent war that will ensue from this decision.